Dicerna Announces Fourth Quarter and Full Year 2014 Financial and Operational Results

-Management to Host Conference Call Today at 4:30 pm ET-

CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Dicerna
Pharmaceuticals, Inc. (NASDAQ: DRNA), a leader in the development of
RNAi-based therapeutics targeting rare inherited diseases involving the
liver and genetically defined cancers, today announced financial and
operational results for the quarter and full year ended December 31,
2014.

"We are pleased with the progress we made in 2014 to advance our key
rare disease and oncology programs. During the year we made major
advances in subcutaneous delivery technology for the liver, laid the
foundation to start clinical development of our lead rare disease
program DCR-PH1 in 2015, and initiated two clinical trials for our
DCR-MYC oncology program," stated Douglas M. Fambrough, Ph.D., Dicerna's
president and chief executive officer. "We remain on track to have
initial clinical data from our DCR-PH1 program and our first Phase 1
DCR-MYC program by year end 2015."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: DsiRNA-EX
Conjugate technologyis a proprietary delivery platform that is
designed to enable convenient subcutaneous delivery for Dicerna's
emerging pipeline of liver-targeted RNAi therapies. These conjugates do
not involve lipid nanoparticles and are built on the DsiRNA-EX platform,
using an extension to one end of the double-stranded DsiRNA molecule.
DsiRNA-EX Conjugates can be delivered subcutaneously while still
retaining capability for intravenous administration, allowing
flexibility in mode of administration.

During its R&D Update on December 15, 2014, Dicerna presented data
from its DsiRNA-EX Conjugate technology and revealed that it is
currently optimizing DsiRNA-EX Conjugates against four targets in the
liver with additional programs planned.

Rare Disease Program Update

DCR-PH1: DCR-PH1 is a DsiRNA-EX-based therapeutic candidate for
Primary Hyperoxaluria 1 (PH1), a rare, inherited disorder of liver
metabolism that usually results in life-threatening damage to the
kidneys. In a genetic mouse model of PH1, Dicerna has shown that DCR-PH1
knocks down the activity of the gene transcript that encodes for the
enzyme glycolate oxidase, thereby significantly reducing the production
of oxalate, the key disease pathology of PH1. This represented a
functional cure for PH1 in the mice.

In October 2014, preclinical data were presented at the 10th Annual
Meeting of the Oligonucleotide Therapeutics Society showing that
DCR-PH1 provides further evidence of the rapid and durable activity of
DCR-PH1, as well as long-term tolerability after four-month chronic
dosing.

In November 2014, Dicerna announced a licensing agreement for the use
of Tekmira's proprietary lipid nanoparticle (LNP) technology for
delivery of DCR-PH1. Tekmira's LNP system has shown in other human
clinical studies to provide potent, safe and effective RNA delivery to
hepatocytes (liver cells). Licensing Tekmira's LNP will streamline the
development path for DCR-PH1.

During its R&D Update on December 15, 2014, Dicerna presented DCR-PH1
data in non-human primates that are supportive of an infrequent dosing
regimen.

Dicerna expects to initiate a Phase 1 trial of DCR-PH1 in 2015, with
initial clinical data released by late 2015.

Oncology Program Update

DCR-MYC: DCR-MYC is a potent and specific inhibitor of MYC, an
oncogene frequently amplified or overexpressed in a wide variety of
cancer tumor types, including hepatocellular carcinoma (HCC). DCR-MYC is
a DsiRNA-based therapeutic formulated in Dicerna's EnCore lipid
nanoparticle for delivery to solid tumors. The MYC oncogene encodes a
small intracellular protein that lacks a good binding site, which makes
it a challenging target for traditional pharmaceutical approaches that
seek to use small molecules or monoclonal antibodies to inhibit protein
activity. Dicerna's RNAi-based approach, which targets gene transcripts
before they are translated into proteins, avoids these difficulties.
This novel approach has yielded encouraging results in pre-clinical
studies, in which Dicerna has shown that DCR-MYC knocks down MYC
transcript activity and significantly reduces tumor volume in multiple
mouse tumor models, including models of HCC.

In April 2014, Dicerna initiated a Phase 1 dose-escalating clinical
study of DCR-MYC in patients with solid tumors, multiple myeloma, or
lymphoma.

Dicerna expects top-line data from this Phase 1 trial in 2015.

In December 2014, Dicerna initiated a Phase 1b/2 clinical trial of
DCR-MYC in patients with advanced HCC. This trial will enroll patients
at sites in the U.S. and Asia. The first patient in this trial was
dosed in January 2015.

Financial Results

Cash Position - As of December 31, 2014, the Company had $98.6
million in cash and cash equivalents and held-to-maturity investments
as compared to $46.6 million in cash and cash equivalents as of
December 31, 2013. In addition, the Company had $1.4 million of
restricted cash, which reflects collateral securing its lease
obligations. In February 2014, the Company completed its initial
public offering (IPO) of common stock, raising net proceeds of
approximately $92.7 million.

R&D Expenses - Research and development (R&D) expenses for
the fourth quarter were $9.9 million, compared to $4.2 million for the
same period in 2013. The increase was due primarily to the initiation
of the clinical trial related to DCR-MYC, an increase in research
activities related to DCR-PH1, which includes $2.5 million related to
license fees paid to Tekmira for the LNP delivery of DCR-PH1, and
increased employee-related expenses, including an increase in
stock-based compensation of $0.8 million. R&D expenses for full year
2014 were $29.5 million, compared to $11.6 million for full year 2013.
The increase was due primarily to the initiation of the clinical trial
related to DCR-MYC, an increase in research activities related to
DCR-PH1, which includes $3.0 million related to license fees paid to
Tekmira for the LNP delivery of DCR-PH1, and increased
employee-related expenses, including an increase in stock-based
compensation of $2.4 million.

G&A Expenses - General and administrative (G&A) expenses
for the fourth quarter were $4.7 million, compared to $2.2 million for
the same period in 2013. The increase was primarily from the increased
costs of operating as a public company and an increase in payroll
related expenses, which includes an increase in stock-based
compensation of $0.5 million. G&A expenses for full year 2014 were
$15.6 million, compared to $5.8 million for full year 2013. The
increase resulted primarily from an increase in payroll related
expenses, which includes an increase in stock-based compensation of
$2.6 million, an increase in the costs of operating as a public
company and an increase in non-employee stock based compensation of
$1.1 million.

Corporate Structure - We have taken several steps to create a
platform to continuously achieve greater operational and financial
efficiencies. Accordingly, we have modified our corporate structure
and, as part of that effort, have transferred our non-U.S.
intellectual property to a non-U.S. wholly-owned subsidiary.

Net Loss Attributable to Common Shareholders - Net loss
attributable to common shareholders for the fourth quarter was $14.6
million compared to a net loss of $6.7 million for the same period in
2013. Net loss attributable to common shareholders for full year 2014
was $48.1 million compared to a net loss of $20.9 million for full
year 2013.

More detailed financial information and analysis may be found in the
Company's Annual Report on Form 10-K, which was filed with the
Securities and Exchange Commission (SEC) on March 12, 2015.

Guidance

Based on our current cash position and operating plan, the Company
re-iterates its expectation that it has sufficient cash to fund
operations through 2016. This estimate assumes no additional funding
from new partnership agreements or debt or equity financing events.

Conference Call

Management will conduct a conference call at 4:30 p.m. ET today to
review the Company's fourth quarter and full year 2014 financial
results. The call can be accessed by dialing (855) 453-3834 or (484)
756-4306 (international), and referencing conference ID 85714307. The
conference call will also be webcast live over the Internet and can be
accessed on the "Events & Presentations" page under the "Investors &
Media" section of the Dicerna Pharmaceuticals website, www.dicerna.com,
prior to the even. A replay of the call will be available beginning at
7:30 p.m. ET on March 12, 2015. To access the replay, please dial
855-859-2056 or 404-537-3406, and refer to conference ID 85714307. The
webcast will also be archived on the Company's website.

About Dicerna Pharmaceuticals, Inc.

Dicerna is a biopharmaceutical company focused on the discovery and
development of innovative treatments for rare, inherited diseases
involving the liver and for cancers that are genetically defined. The
Company is using its proprietary RNA interference (RNAi) technology
platform to build a broad pipeline in these therapeutic areas. In both
rare diseases and oncology, Dicerna is pursuing targets that have
historically been difficult to inhibit using conventional approaches,
but where connections between targets and diseases are well understood
and documented. The Company intends to discover, develop and
commercialize novel therapeutics either on its own or in collaboration
with pharmaceutical partners. For more information, please visit www.dicerna.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. Such
forward-looking statements are subject to risks and uncertainties that
could cause actual results to differ materially from those expressed or
implied in such statements. Applicable risks and uncertainties include
those relating to our preclinical research and other risks identified
under the heading "Risk Factors" included in our most recent Form 10-K
filing and in other future filings with the SEC. The forward-looking
statements contained in this press release reflect Dicerna's current
views with respect to future events, and Dicerna does not undertake and
specifically disclaims any obligation to update any forward-looking
statements.

An itemized reconciliation between net loss on a GAAP basis and
net loss on a non-GAAP basis is as follows:

GAAP net lossattributable to commonstockholders

$

(14,590

)

$

(6,728

)

$

(48,143

)

$

(20,906

)

Adjustments:

Accretion and dividendson redeemable convertible preferredstock

-

9

204

2,388

Preferred stock warrantremeasurement

-

93

2,559

(126

)

Milestone Payment onLicense Agreement

2,500

-

3,000

-

R&D: Stock-basedcompensation

845

92

4,183

124

G&A: Stock-basedcompensation

1,168

250

4,054

371

Non-GAAP net loss

$

(10,077

)

$

(6,284

)

$

(34,143

)

$

(18,149

)

Weighted Average SharesOutstanding -basic anddiluted

17,778,575

33,655

16,070,054

29,463

Numbers may not foot due to rounding

Use of Non-GAAP Financial Measures

We supplement our consolidated financial statements presented on a GAAP
basis by providing additional measures which may be considered
"non-GAAP" financial measures under applicable SEC rules. We believe
that the disclosure of these non-GAAP financial measures provides
additional insight into the ongoing economics of our business and
reflects how we manage our business internally, set operational goals
and forms the basis of our management incentive programs.

These non-GAAP financial measures are not in accordance with generally
accepted accounting principles in the United States and should not be
viewed in isolation or as a substitute for reported, or GAAP, net loss
and net loss per share. Our "Non-GAAP net loss" and "Non-GAAP net loss
per share - basic and diluted" financial measures exclude the following
items from GAAP net loss and net loss per share:

1. Stock-based compensation expense recorded in
accordance with the accounting standard for share-based payments.

We believe that excluding the accounting impact of share-based payments,
for both employees and non-employees, better reflects the recurring
economic characteristics of our business. Share-based payments to
non-employees are measured at each reporting date and recognized as
services are rendered or vesting occurs.

We believe that excluding preferred stock warrant remeasurement better
reflects the recurring economics of our business. Upon our IPO, the
warrants were reclassified to additional paid-in capital and are no
longer marked to market.

3. Milestone Payment on License Agreement.

We believe that excluding the payment to Tekmira for the license to
their LNP delivery technology for use in our PH1 development program
better reflects the recurring nature of our business. Based on our
current drug development program and recent advances in our technology
platform, we do not expect to enter into similar licensing arrangements.

4. Other items.

We evaluate other items on an individual basis, and consider both the
quantitative and qualitative aspects of the item, including (i) its size
and nature, (ii) whether or not it relates to our ongoing business
operations, and (iii) whether or not we expect it to occur as part of
our normal business on a regular basis.